Análisis de las características clínicas y epidemiológicas principales de los pacientes que utilizan hormona de crecimiento atendidos en el servicio de endocrinología en el Hospital Nacional de Niños, en el período comprendido entre enero 2008 y diciembre 2023
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Abstract
Antecedentes: El tratamiento con hormona de crecimiento recombinante (rhGH) se utiliza en el Hospital Nacional de Niños desde 1987. El único análisis previo incluyó 295 pacientes entre 2008 y 2015, de los cuales solo el 18.3 % había completado el tratamiento. Este estudio tiene como objetivo analizar las características clínicas y epidemiológicas de los pacientes tratados con rhGH entre 2008 y 2023, así como evaluar la efectividad del tratamiento en población pediátrica costarricense.
Métodos: Se realizó un estudio observacional, descriptivo y retrospectivo con 672 pacientes tratados en la consulta de Endocrinología del Hospital Nacional de Niños. Se recopilaron variables clínicas, antropométricas, bioquímicas y relacionadas al tratamiento. Se analizaron diferencias por diagnóstico y estado del tratamiento (activo, completo o suspendido) y se identificaron predictores de respuesta satisfactoria, definida como alcanzar una talla final ≤2 DE por debajo del potencial genético. Se aplicaron pruebas no paramétricas, chi-cuadrado y regresión logística binaria.
Resultados: El diagnóstico más frecuente fue déficit de GH (63.1%), seguido por síndrome de Turner (16.8%), PEG (14.4%) y otros (5.7%). La mediana de edad al inicio fue de 107.5 meses, más baja en PEG (69 meses), que también presentó el Z-score inicial más comprometido (−3.2). El 43% completó el tratamiento, 36.9% continuaba activo y 20.1% lo había abandonado. El cumplimiento fue significativamente menor en los abandonos (45.9%, p<0.001). De los 289 pacientes que completaron el tratamiento, el 52.2% alcanzó una talla final satisfactoria. Los predictores significativos fueron sexo masculino (OR 3.0, p=0.013), cumplimiento regular (OR 6.08, p=0.010), mayor Z-score de talla inicial (OR 4.48, p<0.001), mejor potencial genético (OR 2.39, p=0.018) y menor pico de GH (OR 0.77, p=0.003). Se reportaron efectos adversos en el 9.1%, sin relación con la dosis ni el diagnóstico.
Conclusiones: Los hallazgos concuerdan con registros internacionales como KIGS, GeNeSIS y NordiNet, y destacan la importancia del diagnóstico temprano, la adherencia sostenida y la adecuada selección diagnóstica para optimizar los resultados del tratamiento con hormona de crecimiento.
Background: Recombinant human growth hormone therapy has been used at the Hospital Nacional de Niños in Costa Rica since 1987. The only previous analysis included 295 patients between 2008 and 2015, of whom only 18.3% had completed treatment. This study aims to analyze the clinical and epidemiological characteristics of patients treated with rhGH from 2008 to 2023, and to evaluate treatment effectiveness in the Costa Rican pediatric population. Methods: An observational, descriptive, and retrospective study was conducted including 672 patients treated in the Endocrinology outpatient clinic at the National Children's Hospital. Clinical, anthropometric, biochemical, and treatment-related variables were collected. Differences were analyzed according to diagnosis and treatment status (active, completed, or discontinued), and predictors of a satisfactory response; defined as achieving a final height within ≤2 SD below the genetic target, were identified. Nonparametric tests, chi-square, and binary logistic regression were applied. Results: The most frequent diagnosis was GH deficiency (63.1%), followed by Turner syndrome (16.8%), small for gestational age (SGA) (14.4%), and other diagnoses (5.7%). The median age at treatment initiation was 107.5 months, lower in SGA patients (69 months), who also had the most compromised initial height Z-score (−3.2). Of the total, 43% completed treatment, 36.9% remained on active therapy, and 20.1% had discontinued. Treatment adherence was significantly lower among those who discontinued (45.9%, p<0.001). Among the 289 patients who completed treatment, 52.2% achieved a satisfactory final height. Significant predictors of treatment success included male sex (OR 3.0, p=0.013), regular adherence (OR 6.08, p=0.010), higher initial height Z-score (OR 4.48, p<0.001), better genetic height potential (OR 2.39, p=0.018), and lower GH peak (OR 0.77, p=0.003). Adverse events were reported in 9.1% of patients, with no association with GH dose or diagnosis. Conclusions: These findings are consistent with international registries such as KIGS, GeNeSIS, and NordiNet, and emphasize the importance of early diagnosis, sustained adherence, and appropriate diagnostic selection to optimize treatment outcomes with rhGH.
Background: Recombinant human growth hormone therapy has been used at the Hospital Nacional de Niños in Costa Rica since 1987. The only previous analysis included 295 patients between 2008 and 2015, of whom only 18.3% had completed treatment. This study aims to analyze the clinical and epidemiological characteristics of patients treated with rhGH from 2008 to 2023, and to evaluate treatment effectiveness in the Costa Rican pediatric population. Methods: An observational, descriptive, and retrospective study was conducted including 672 patients treated in the Endocrinology outpatient clinic at the National Children's Hospital. Clinical, anthropometric, biochemical, and treatment-related variables were collected. Differences were analyzed according to diagnosis and treatment status (active, completed, or discontinued), and predictors of a satisfactory response; defined as achieving a final height within ≤2 SD below the genetic target, were identified. Nonparametric tests, chi-square, and binary logistic regression were applied. Results: The most frequent diagnosis was GH deficiency (63.1%), followed by Turner syndrome (16.8%), small for gestational age (SGA) (14.4%), and other diagnoses (5.7%). The median age at treatment initiation was 107.5 months, lower in SGA patients (69 months), who also had the most compromised initial height Z-score (−3.2). Of the total, 43% completed treatment, 36.9% remained on active therapy, and 20.1% had discontinued. Treatment adherence was significantly lower among those who discontinued (45.9%, p<0.001). Among the 289 patients who completed treatment, 52.2% achieved a satisfactory final height. Significant predictors of treatment success included male sex (OR 3.0, p=0.013), regular adherence (OR 6.08, p=0.010), higher initial height Z-score (OR 4.48, p<0.001), better genetic height potential (OR 2.39, p=0.018), and lower GH peak (OR 0.77, p=0.003). Adverse events were reported in 9.1% of patients, with no association with GH dose or diagnosis. Conclusions: These findings are consistent with international registries such as KIGS, GeNeSIS, and NordiNet, and emphasize the importance of early diagnosis, sustained adherence, and appropriate diagnostic selection to optimize treatment outcomes with rhGH.
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Endocrinología, tratamiento médico